Thryv Therapeutics' LQT-1213 Receives FDA Orphan Drug Status for Long QT Syndrome

MONTREAL, Oct. 1, 2024 /PRNewswire/ - Thryv Therapeutics, a clinical stage biotech company focused on developing treatments for rare cardiovascular diseases, has announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to LQT-1213 for treating Long QT Syndrome (LQTS). LQT-1213, a novel SGK1 inhibitor, is specifically designed to address congenital LQTS. Thryv has created a series of SGK1 inhibitors targeting cardiometabolic stress in various arrhythmic conditions, including LQTS, atrial fibrillation, and heart failure.

Debra Odink, PhD, President and Chief Development Officer of Thryv Therapeutics, expressed satisfaction with the FDA's decision. "People with Long QT Syndrome deserve a thoroughly researched and FDA-approved treatment for combating this potentially fatal genetic disease," Odink stated. "Currently, no FDA-approved therapies exist for Long QT Syndrome, and this designation underscores the potential of LQT-1213 to meet this unmet need. It also provides critical incentives for Thryv to advance our development of pivotal efficacy studies for congenital Long QT Syndrome."

Following the FDA's request, clinical data from Thryv's ongoing Wave I clinical study involving patients with congenital LQTS was submitted to the Office of Orphan Products Designation. Shortly after, the company received confirmation that its request for Orphan Drug Designation had been approved.

The FDA's Orphan Drug Designation is granted to drugs and biologics aimed at treating rare diseases affecting fewer than 200,000 people in the United States. This designation offers several advantages to support the development of treatments for rare conditions, such as tax credits for clinical trial expenses, exemption from certain FDA fees, and up to seven years of market exclusivity post-approval.

About LQT-1213
LQT-1213 is a powerful and selective serum glucocorticoid regulated kinase 1 (SGK1) inhibitor under development as an investigational treatment for Long QT Syndrome Types 1, 2, and 3. By targeting SGK1, LQT-1213 aims to shorten the extended QTc interval in individuals with LQTS, thereby reducing the risk of life-threatening arrhythmias. Currently, LQT-1213 is being evaluated in clinical trials for its effectiveness and safety in treating congenital LQTS.

About Thryv Therapeutics Inc.
Thryv Therapeutics Inc. is a privately held company based in Montreal, Quebec, Canada. The company is pioneering a precision medicine approach to develop highly selective SGK1 inhibitors for treating Long QT Syndrome, atrial fibrillation, and heart failure. 

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